Chinese researchers become first to test CRISPR gene editing in humans, using immune cells to attack cancer

CRISPR-Cas9 technology uses "molecular scissors" to cut and splice DNA, as shown in this graphic. (Credit: Berkeley Lab)
CRISPR-Cas9 technology uses “molecular scissors” to cut and splice DNA, as shown in this graphic. Photo: Berkeley Lab.

Researchers at Sichuan University in Chengdu, China, have injected the first CRISPR-edited cells into a patient. The procedure is part of a study designed to test the technique’s safety for use in humans, according to a report from Nature.

The team, led by oncologist Lu You, used CRISPR gene editing to alter a patient’s immune cells so the cells can identify and attack cancer. The researchers plan to inject 10 study patients with edited immune cells to test the safety of the approach.

Lu and his team edged out a similar study in the US, which was given the green light this summer and is set to begin early in 2017. That study will take place at the University of Pennsylvania, and will also test the safety of CRISPR-assisted immunotherapy in a small batch of patients.

Dr. David Maloney, medical director of the Bezos Family Immunotherapy Clinic, in the facility. The Clinic is the first of its kind and solely geared towards researching immunotherapy treatments for advanced cancers. Photo: Fred Hutchinson Cancer Research Center.
Dr. David Maloney, medical director of the Bezos Family Immunotherapy Clinic at the Fred Hutchinson Cancer Research Center in Seattle. Photo: Fred Hutch.

Carl June, an advisor to the planned U.S. trial and an immunotherapy specialist at the University of Pennsylvania, told Nature that news of the Chinese study could be the beginning of a space-race like competition between the U.S. and China for dominance in gene editing.

“I think this is going to trigger ‘Sputnik 2.0’, a biomedical duel on progress between China and the United States, which is important since competition usually improves the end product,” he said.

Immunotherapy research is an important part of Seattle’s biotech ecosystem. Fred Hutchinson Cancer Research Center was an early pioneer in the technique, and recently opened an immunotherapy research clinic to expand the scope of their immunotherapy trials.

Several Seattle-area companies are also involved in immunotherapy development, notably Fred Hutch spin-off Juno Therapeutics. Much of the research in the Seattle area has been on CAR T-Cell therapies, which program a patient’s immune cells to grow receptors that recognize and attack cancer cells.

No CRISPR studies are planned in the Seattle area for the time being.

Lu and his team used the CRISPR-CAS9, an agent that combines a DNA-cutting enzyme with a molecular guide that tells the enzyme what to cut out of a cell’s DNA. The tool can be programmed to cut out certain genes or sections of genes — in this case, removing genes that control the PD-1 protein, which is like a set of brakes for the immune system.

The hope is that removing this gene will help the immune cells fight cancerous cells and eventually cure a patient of the disease.


Post Author: Tech Review